Novo Nordisk has announced new phase 3 data showing that investigational denecimig (Mim8) continued to demonstrate long-term safety and efficacy in children, adolescents, and adults living with hemophilia A, including people with and without inhibitors.
The latest findings from the FRONTIER4 extension study highlight denecimig’s potential as a preventive treatment option across multiple dosing schedules, including once-monthly, once-every-two-weeks, and once-weekly administration.
"The positive safety and efficacy findings from FRONTIER4 reinforce denecimig’s potential as a preventive treatment option for children, adolescents, and adults with hemophilia A, regardless of their inhibitor status or the dosing frequency used,” said Martin Holst Lange, chief scientific officer and executive vice president, Research & Development at Novo Nordisk.
“The breadth and totality of our data presented at ISTH reflect Novo Nordisk’s commitment to advancing treatment innovation to address the diverse needs of people living with hemophilia.”
Interim results from the open-label, phase 3 FRONTIER4 long-term study included 426 participants aged 1 year and older receiving denecimig prophylaxis — 365 adults and adolescents with a median observation period of 0.50 years and 61 children with a median observation period of 0.33 years.
The safety profile remained consistent with previous findings across the FRONTIER clinical program. Injection-site reactions were uncommon, occurring in 2.0% of injections among children and 1.8% of injections among adolescents and adults. All reported reactions were mild and transient, with no clinical evidence of neutralizing antibodies observed.
Efficacy results also remained consistent across all evaluated dosing regimens and regardless of inhibitor status. Estimated mean annualized bleeding rates (ABRs) were 0.75 for adults and adolescents and 0.37 for children.
Across dosing groups, approximately 71% of adults and adolescents and 89% of children experienced zero treated bleeds while receiving denecimig.
Patient-reported outcomes from FRONTIER4 further showed sustained improvements seen in earlier FRONTIER trials. Participants aged 12 and older reported improvements in joint pain, while people across age groups reported reduced treatment burden.
The majority of participants also rated the denecimig pen-injector positively, with 94.1% of 185 participants describing it as easy or very easy to use and 89.7% reporting it was quick or very quick to prepare and inject.
Additional post hoc analyses from the phase 3 FRONTIER2 and FRONTIER5 studies found that denecimig prophylaxis increased thrombin generation — a measure of the body’s ability to form clots — into the normal reference range in adolescents and adults without excessive response, providing further support for the treatment’s clinical profile.
Novo Nordisk submitted denecimig for review by the US Food and Drug Administration (FDA) in September 2025 through a Biologics License Application (BLA).
“When managing a chronic condition like hemophilia A, it's important that treatments are evaluated over the long-term and also offer dosing optionality,” said Guy Young, Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles.
“These data suggest denecimig, which has delivered consistent results across the entire FRONTIER program, has the potential to have a truly meaningful impact for a diverse array of people with hemophilia A.”
